September 2, 2026

Teva TEV-408 Celiac Phase 2a Wins, Simcere-Roche $1.53B SIM0660 Deal, Inventiva NATiV3 Enrollment Done, FDA CBER Pilot

Teva anti-IL-15 hits celiac Phase 2a endpoint, Simcere-Roche sign $1.53B tri-specific antibody deal, Inventiva NATiV3 MASH data on track for Q4.

10 stories · Covering clinical-trials, industry, regulatory

Editor's Note

Wednesday delivered a modest Phase 2 win in a stubborn autoimmune indication, a $1.53 billion China-to-Roche antibody licensing deal, and a clean marker on the last patient visit for a Phase 3 MASH readout that arrives at the end of the year. Teva Pharmaceutical Industries's TEV-408 (an anti-IL-15 monoclonal antibody) hit its primary endpoint in the Phase 2a celiac disease study, showing statistically significant prevention of gluten-induced intestinal damage at Week 8. Simcere Zaiming, a subsidiary of Simcere Pharmaceutical Group, signed an exclusive global licensing agreement with Roche for SIM0660 (a CD79a/CD19/CD3 tri-specific antibody) at $75 million upfront plus up to $1.53 billion in aggregate milestones — one more entry in the China-to-multinational T-cell-engager pipeline that continues to feed Big Pharma's B-cell-mediated disease portfolios. Inventiva completed the last patient last visit in the Phase 3 NATiV3 study of lanifibranor in MASH with F2/F3 fibrosis (1,009 patients in the main cohort), with topline results expected in Q4 2026 and NDA filing in H1 2027. On the peptide-adjacent regulatory calendar, the CBER Phase 1 fast-track IND pilot opens applications in September 2026, and three September PDUFA dates set up further catalysts: Ultragenyx UX111 gene therapy for Sanfilippo Type A on September 19, Merck Winrevair (sotatercept-csrk) PAH label expansion on September 21, and Ionis zilganersen antisense oligonucleotide for Alexander disease on September 22.

Clinical Trials

Teva TEV-408 Anti-IL-15 Monoclonal Antibody Hits Primary Endpoint in Phase 2a Celiac Disease Study; 27-Point IEL Density Advantage Over Placebo at Week 8

Teva Pharmaceutical Industries (NYSE: TEVA) announced Wednesday September 2, 2026 positive topline results from the ongoing Phase 2a study of TEV-408 (an investigational anti-interleukin-15 monoclonal antibody) in adults with celiac disease already following a gluten-free diet. The 50-patient study met its primary endpoint by demonstrating statistically significant and clinically substantive prevention of gluten-induced intestinal damage versus placebo at Week 8. Key efficacy: LS mean change from baseline in intraepithelial lymphocyte (IEL) density was +27.60 for placebo versus +0.37 for TEV-408 (treatment difference -27.23, 95% CI: -39.67 to -14.79). TEV-408 also demonstrated lower gastrointestinal symptom scores versus placebo on the patient-reported Celiac Disease Symptom Diary. Safety was clean with no signals observed. TEV-408 received FDA Fast Track designation in May 2025 and represents a novel mechanism in celiac disease where no disease-modifying therapies are approved and standard-of-care remains strict gluten-free diet adherence. Teva positioned the win as further validation of its 'pipeline-in-a-product' anti-IL-15 platform strategy.

#teva #tev-408 #anti-il-15 #celiac-disease #phase-2a #gluten-induced-intestinal-damage #iel-density #csdd-patient-reported #fast-track-designation #50-patient-study #non-peptide

Industry

Simcere Zaiming Signs Exclusive Global Licensing Agreement With Roche for SIM0660 CD79a/CD19/CD3 Tri-Specific Antibody; $75 Million Upfront Plus Up to $1.53 Billion in Milestones

Simcere Zaiming (a subsidiary of China's Simcere Pharmaceutical Group) announced Wednesday September 2, 2026 an exclusive global licensing agreement with Roche for SIM0660, a T-cell engager tri-specific antibody targeting CD79a, CD19, and CD3 for B-cell-mediated diseases. Deal terms: $75 million upfront plus up to $1.53 billion in total development, regulatory, and commercial milestone payments plus tiered royalties up to double-digits on future net sales. Roche acquires exclusive global rights to develop, manufacture, and commercialize SIM0660. The molecule combines a CD3-engaging arm with binding domains targeting the two B-cell antigens CD79a and CD19 to induce T-cell-mediated cytotoxicity while limiting cytokine release, positioned to compete with the emerging poly-specific antibody category (Amgen tarlatamab, Regeneron REGN5459, Janssen amivantamab). The transaction is the latest in a $60+ billion H1 2026 wave of China-to-multinational biotech licensing that continues to feed U.S. and European pharma pipelines.

#simcere-zaiming #simcere-pharmaceutical-group #roche #sim0660 #cd79a-cd19-cd3 #tri-specific-antibody #t-cell-engager #b-cell-mediated-diseases #75-million-upfront #1-53-billion-milestones #china-to-multinational-licensing #non-peptide

Clinical Trials

Inventiva Completes Last Patient Last Visit in Phase 3 NATiV3 Trial of Lanifibranor in MASH; Topline Data Expected Q4 2026 With NDA H1 2027

Inventiva SA (NASDAQ: IVA) announced Wednesday September 2, 2026 completion of the last patient's final 72-week visit in the Phase 3 NATiV3 study of lanifibranor (a pan-PPAR α/δ/γ agonist) in adults with biopsy-proven non-cirrhotic MASH and F2/F3 fibrosis. Enrollment: 1,009 patients in the main cohort plus an additional 410 patients in the exploratory cohort. NATiV3 assesses lanifibranor on histological endpoints including MASH resolution and improvement of fibrosis of at least one stage after 72 weeks of treatment. Inventiva expects to report topline data in Q4 2026 with NDA filing planned for H1 2027. Lanifibranor differentiates from the standard-of-care Madrigal Rezdiffra (resmetirom, THR-β agonist, approved March 2024) plus the emerging Boehringer Ingelheim survodutide, Novo Nordisk semaglutide MASH, and Akero Therapeutics efruxifermin (FGF21) programs by engaging three complementary PPAR isoforms in parallel to address steatosis, inflammation, and fibrosis.

#inventiva #iva #lanifibranor #nativ3 #pan-ppar-agonist #mash #f2-f3-fibrosis #1009-patients #topline-q4-2026 #nda-h1-2027 #rezdiffra-comparator #resmetirom #non-peptide

Industry

Myasthenia Gravis Global 7MM Market Forecast to Grow at 11.1% CAGR From $4.5 Billion in 2024 to $13.0 Billion in 2034 Per GlobalData

Analytics firm GlobalData published Wednesday September 2, 2026 a myasthenia gravis (MG) market forecast projecting the seven-major-markets (7MM: United States, France, Germany, Italy, Spain, United Kingdom, Japan) MG treatment market to grow from $4.5 billion in 2024 to $13.0 billion in 2034 at an 11.1% CAGR, driven by launches and label expansions in the anti-FcRn class (argenx Vyvgart Hytrulo, Johnson & Johnson Imaavy nipocalimab), complement inhibitors (Alexion/AstraZeneca Ultomiris, Soliris), and BAFF/BLyS-targeting therapies (Cytokinetics aficamten in adjacent indications). The forecast supports the ongoing peptide-therapeutic case in autoimmune indications where synthetic peptide-vaccine and peptide-immunomodulator approaches (thymalfasin, thymosin analogs) are being evaluated as adjuncts to the biologics-first standard-of-care. Anti-FcRn represents the fastest-growing MG segment given the manageable safety profile and infrequent dosing versus the plasmapheresis and IVIG protocols that dominated the pre-Vyvgart era.

#myasthenia-gravis #globaldata #7mm-market-forecast #13-billion-by-2034 #11-1-percent-cagr #anti-fcrn #vyvgart #imaavy #argenx #johnson-johnson #ultomiris #peptide-immunomodulator #non-peptide

Regulatory

FDA CBER Phase 1 Fast-Track IND Pilot Opens Applications in September 2026; Up to 10 Programs Selected by Year End

The FDA Center for Biologics Evaluation and Research (CBER) confirmed Wednesday September 2, 2026 that the previously-announced Phase 1 fast-track IND pilot program is opening applications during September 2026, with up to 10 investigational programs selected by year end. The pilot follows the Center for Drug Evaluation and Research (CDER) parallel pilot announced earlier in 2026 and is designed to accelerate first-in-human clinical trial initiation for high-priority biologics (including cell and gene therapies, therapeutic vaccines, peptide-based biologics, and blood-derived products). The pilot matters for the peptide-industry pipeline because peptide-conjugate cancer vaccines, therapeutic peptide vaccines targeting infectious disease, and peptide-antimicrobial biologics all sit within the CBER regulatory scope and would benefit from the accelerated review timeline. Selection criteria emphasize unmet medical need, novel mechanism, and preclinical data quality; individual selection results will not be public.

#fda-cber #phase-1-fast-track-pilot #ind-expedite #up-to-10-programs #cell-and-gene-therapy #therapeutic-vaccines #peptide-biologics #cder-parallel-pilot #high-priority-biologics

Regulatory

Ultragenyx UX111 AAV9 Gene Therapy for Sanfilippo Syndrome Type A PDUFA Date September 19, 2026

Ultragenyx Pharmaceutical (NASDAQ: RARE) awaits its September 19, 2026 PDUFA target action date for UX111 (rebisufligene etisparvovec, an AAV9 gene therapy delivering the SGSH gene) for the treatment of Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA, MPS IIIA, a rare autosomal recessive lysosomal storage disorder caused by SGSH gene mutations that results in progressive cognitive decline in early childhood and death typically by the second decade). If approved, UX111 would become the first FDA-approved therapy for MPS IIIA, where the current standard of care remains supportive symptom management. The AAV9-based intravenous delivery approach mirrors the Novartis Zolgensma (onasemnogene abeparvovec) commercial template for spinal muscular atrophy and continues the neurometabolic gene therapy pipeline. Not a peptide, but the approval would extend the AAV gene therapy commercial category that has continued to reshape the rare-disease landscape alongside enzyme replacement therapies and the emerging small-molecule chaperone class.

#ultragenyx #rare #ux111 #rebisufligene-etisparvovec #aav9-gene-therapy #sanfilippo-syndrome #mps-iiia #sgsh-gene #september-19-pdufa #first-in-indication #zolgensma-comparator #non-peptide

Regulatory

Merck Winrevair (Sotatercept-csrk) Pulmonary Arterial Hypertension Label Expansion PDUFA Target Date September 21, 2026

Merck (NYSE: MRK) awaits its September 21, 2026 PDUFA target action date for a further label expansion of Winrevair (sotatercept-csrk, an activin signaling inhibitor administered as subcutaneous injection every three weeks) in pulmonary arterial hypertension (PAH). Winrevair was originally FDA-approved March 26, 2024 for adults with PAH (WHO Group 1) to increase exercise capacity, improve WHO functional class, and reduce risk of clinical worsening events, based on the Phase 3 STELLAR trial. The pending label expansion is expected to cover an earlier line of therapy and/or a broader patient population based on the ZENITH and HYPERION Phase 3 trials that reported earlier in 2026 (ZENITH: PAH high-risk patients on maximal background therapy; HYPERION: newly diagnosed PAH). The Winrevair franchise reached $260 million in Q2 2026 revenue and is on track to reach $1 billion annual run rate before end of 2026. Sotatercept-csrk is a fusion protein of activin receptor type IIA and the Fc portion of human immunoglobulin.

#merck #mrk #winrevair #sotatercept-csrk #activin-signaling-inhibitor #pulmonary-arterial-hypertension #pah #september-21-pdufa #zenith-trial #hyperion-trial #stellar-original-approval #260-million-q2-2026 #protein-therapeutic

Regulatory

Ionis Zilganersen Antisense Oligonucleotide for Alexander Disease PDUFA Target Date September 22, 2026

Ionis Pharmaceuticals (NASDAQ: IONS) awaits its September 22, 2026 PDUFA target action date for zilganersen (an intrathecally-administered antisense oligonucleotide designed to reduce production of glial fibrillary acidic protein / GFAP) for the treatment of Alexander disease, a rare autosomal dominant neurodegenerative disease caused by GFAP gene mutations. If approved, zilganersen would become the first FDA-approved therapy for Alexander disease and Ionis's next commercial launch following Wainua (eplontersen) for hereditary transthyretin amyloid polyneuropathy (with the CARDIO-TTransform ATTR-CM primary endpoint miss now behind the company). Zilganersen was granted FDA Fast Track designation and Orphan Drug designation. Alexander disease presents in infancy through adulthood with progressive motor and cognitive decline; no disease-modifying therapies are currently approved. Not a peptide, but the antisense oligonucleotide class continues to complement peptide-based rare-disease therapies (Rein Therapeutics LTI-03 Caveolin-1 peptide for IPF among others) as the RNA modality expands into ultra-orphan indications.

#ionis-pharmaceuticals #ions #zilganersen #antisense-oligonucleotide #gfap-targeting #alexander-disease #intrathecal-administration #september-22-pdufa #first-in-indication #wainua-eplontersen-context #orphan-drug-fast-track #non-peptide

Clinical Trials

Ascletis ASC36 Amylin Receptor Peptide Agonist Once-Monthly to Once-Quarterly Phase 1 Enrollment Continues in U.S. Obesity Study

Ascletis Pharma (HKEX: 1672) continued enrollment through early September 2026 in the two U.S. Phase 1 studies of ASC36 (a once-monthly to once-quarterly subcutaneous amylin receptor peptide agonist for obesity) and ASC36_35FDC (a once-monthly subcutaneous fixed-dose combination of ASC36 plus GLP-1R/GIPR peptide agonist ASC35) initiated August 10, 2026 following FDA IND clearance July 5. In head-to-head diet-induced-obesity rat studies, ASC36 monotherapy demonstrated approximately 91% greater relative body weight reduction versus Zealand-Roche's petrelintide and approximately 32% greater versus Eli Lilly's eloralintide. If the Phase 1 tolerability profile confirms preclinical differentiation, ASC36 would enter the amylin analog Phase 2 field alongside Novo Nordisk's cagrilintide-in-CagriSema, AstraZeneca's AZD6234 (Rome EASD 2026 September 28-October 2 readout), Metsera's MET-233i, and the Roche-Zealand petrelintide-plus-enicepatide combination Phase 2 initiation planned mid-2026.

#ascletis-pharma #asc36 #asc35 #asc36-35fdc #amylin-receptor-peptide-agonist #once-monthly-to-quarterly #obesity #phase-1-us #petrelintide-comparator #eloralintide-comparator #azd6234-comparator #met-233i-comparator

Industry

Novartis Reports 4%+ Share Gain Following Tuesday REMODEL RMS Phase 3 Win; Focus Turns to Pacibekitug IL-6 Phase 3 Advance and Avidity RNAi Neuromuscular Integration

Novartis (NYSE: NVS) shares rose more than 4% Tuesday September 1 and continued climbing Wednesday September 2, 2026 following the twin REMODEL-1 and REMODEL-2 Phase 3 wins of remibrutinib (an oral BTK inhibitor) in relapsing multiple sclerosis, which met both primary endpoints versus teriflunomide with a clean liver-safety profile across the 4,500+ patient program. Investor focus now turns to two additional Novartis growth-vehicle programs: pacibekitug (an anti-IL-6 monoclonal antibody acquired in the $1.4 billion Tourmaline Bio acquisition earlier in 2026) advancing into Phase 3 for cardiovascular risk reduction despite Novo Nordisk's ZEUS ziltivekimab Phase 3 miss at ESC 2026 last weekend; and the ongoing integration of the $12 billion Avidity Biosciences acquisition centered on RNAi drug delivery to muscle tissue for facioscapulohumeral muscular dystrophy, DM1 myotonic dystrophy, and Duchenne muscular dystrophy programs. Novartis Q3 2026 earnings release is scheduled for late October.

#novartis #nvs #remibrutinib #remodel-follow-through #pacibekitug #tourmaline-bio #1-4-billion-acquisition #avidity-biosciences #12-billion-acquisition #rnai-neuromuscular #cardiovascular-il-6 #share-gain-4-percent #non-peptide